two pivotal trials have shown that combining Kalydeco with a second Vertex drug called lumacaftor (VX-809) - is effective in treating patients aged 12 or more with two copies of the F508del ...

NHS England has agreed a deal with Vertex for its three-drug cystic fibrosis therapy Kaftrio ... 12 or over who have two copies of the F508del mutation gene, or one copy of F508del and a minimal ...

In two new papers, researchers from The Jackson Laboratory (JAX) report the successful use of two approaches—gene therapy and ...

The drugmaker sees additional growth opportunities in ophthalmology and neuromuscular disorders, Chief Medical Officer ...

Credit: Jo Panuwat D/Shutterstock. Genprex has dosed the first subject in the Phase II expansion portion of its Acclaim-3 trial of the Reqorsa gene therapy (quaratusugene ozeplasmid) for ...

Patients with metastatic colorectal cancer (mCRC) harboring BRAF V600E mutations benefited from first-line treatment with the ...

Scientists have discovered a potential new treatment for familial hypercholesterolemia, inspired by signs seen in the Mona ...

FDA 2025 pulmonology drug alerts, including drug approvals/warnings, vaccine approvals, and medical device recalls/updates.

An in vivo base editing approach targeting the PRNP gene led to a 52% increase in the lifespan of mouse models inoculated with the most common sporadic and genetic types of human pathogenic prion ...

The FDA approved a wide range of therapies in 2024, including many new molecular entities and biological products.

Jammu-Srinagar Vande Bharat: Launched as a part of India's 'Make in India' initiative, the Vande Bharat Express is India's first indigenous semi-high-speed train. (Image: PTI) Built to endure the ...

It markets TRIKAFTA/KAFTRIO for people with CF with at least one F508del mutation for 2 years ... which is in Phase 1 clinical trial; VX-548, a non-opioid medicine for the treatment of acute ...